Gene therapy - MB140P93
Title: Genová terapie
Czech title: Genová terapie
Guaranteed by: Department of Genetics and Microbiology (31-140)
Faculty: Faculty of Science
Actual: from 2021
Semester: winter
E-Credits: 3
Examination process: winter s.:combined
Hours per week, examination: winter s.:2/0, Ex [HT]
Capacity: unlimited
Min. number of students: 5
4EU+: no
Virtual mobility / capacity: no
State of the course: taught
Language: Czech
Level: specialized
Note: enabled for web enrollment
Guarantor: RNDr. Michal Šmahel, Ph.D.
Teacher(s): RNDr. Michal Šmahel, Ph.D.
Opinion survey results   Examination dates   WS schedule   
Annotation -
Gene therapy is a discipline striving for the application of up-to-date knowledge and methods into a clinical practice. It comprises methodological approaches of molecular biology and genetic engineering and also understandings from genetics, immunology, biochemistry, virology, oncology, and other biological and medical fields. In a general part of the course, principles, mechanisms, and potential usage of gene therapy will be explained as well as gene transfer and ethical problems associated with gene therapy. Another part of the course will be focused on the utilization of gene therapy in oncology and the description of main anti-tumor therapeutic procedures. Finally, students will prepare short presentations (about 10 min) about the development of gene therapy for selected non-malignant diseases.
Last update: Šmahel Michal, RNDr., Ph.D. (01.11.2021)
Literature -

Mauro Giacca: Gene Therapy, Springer-Verlag Mailand, 2010

  eBook ISBN    978-88-470-1643-9,  Hardcover ISBN    978-88-470-1642-2,  Softcover ISBN    978-88-470-5555-1,  DOI    10.1007/978-88-470-1643-9


Wolfgang Walther (ed.): Current Strategies in Cancer Gene Therapy, Springer International Publishing, 2016

  Hardcover ISBN     978-3-319-42932-8,    eBook ISBN     978-3-319-42934-2,    DOI 10.1007/978-3-319-42934-2

Last update: Schierová Michaela, RNDr., Ph.D. (24.10.2019)
Requirements to the exam -

Outline of student presentations

1. Disease characterization and frequency.

2. Standard therapy and its efficacy.

3. Therapeutic gene/genetic material (including regulatory sequences)

4. Delivery methods

5. Preclinical examination (model)

6. Clinical trials


This presentation is a prerequisite for examination.



A written test composed of 50 questions (20 multiple choice questions, 20 questions requiring short answers).

Last update: Šmahel Michal, RNDr., Ph.D. (26.07.2023)
Syllabus -

I. General part
Principles, mechanisms and possibilities of gene therapy; ethical problems
Types of therapeutic nucleic acids (NA)
   - genes encoding proteins 
   - non-coding NA (oligonucleotides, catalytic NA, regulatory NA, aptamers)
Delivery of genetic material
   - viral (retroviruses, adenoviruses, herpetic viruses, adeno-associated viruses)
   - non-viral - physical (electroporation, sonoporation, biolistic delivery)
                   - chemical (liposomes, polymers)
Gene editing

II. Gene therapy of tumors
Regulation of gene expression
   - modified T lymphocytes (TCR, CAR)
   - vaccines - DNA and RNA vaccines, recombinant viruses, cell vaccines
Suicide genes
Oncolytic viruses

III. Gene therapy of non-malignant diseases – student presentations
Hematopoietic stem cells
Neurodegenerative diseases
Muscle dystrophy
Cardiac diseases
Viral infections
Ocular diseases
Liver diseases
Lung diseases


Last update: Šmahel Michal, RNDr., Ph.D. (24.10.2019)